This animation shows how mutations in an ion channel protein lead to the genetic disease cystic fibrosis. The animation also discusses how research on this protein has been used to develop treatments for the disease.

This video describes the case of a patient with retinitis pigmentosa (RP), a progressive disease that leads to blindness, and how physician-scientist Dr. Ed Stone approached the search for the causal mutation.

This film describes the scientific principles and the research efforts involved in the development of a gene therapy for a congenital form of blindness, and how a young patient benefited from this medical breakthrough.